Plan Would Hasten OK for Certain New Drugs : Health: They would be sold before effectiveness is proved. The focus is on treatments for fatal illnesses.
Federal health officials are considering the possibility of creating a new drug approval category that would allow certain drugs to be sold before they have been thoroughly studied, but which would require close scrutiny after they reach the marketplace, The Times has learned.
Dr. David Kessler, new commissioner of the Food and Drug Administration, has established an internal task force to examine the idea of “conditional” approval of drugs for life-threatening illnesses, such as AIDS and cancer, where there are few or no therapeutic alternatives, FDA officials confirmed Friday.
If put in effect, this would be the most far-reaching step in the government’s recent efforts to make such drugs more quickly available to the public. Federal health officials, prompted in part by the AIDS epidemic, already have allowed the widespread use of several experimental AIDS drugs--through parallel track and other programs--but only under tightly controlled circumstances.
Currently, most drugs are first studied in a small group of people to determine if they are safe--that is, to see whether the drugs produce toxic effects. Then they are studied in several phases to see whether they work before they are allowed to be sold. They are difficult to remove once they are in the marketplace.
The new proposal would allow certain drugs to be sold once they are proven safe, but before it is determined whether they work. However, it would be easier for the federal government to pull the drugs off the market if problems arose later. Presumably, post-market surveillance would be conducted by the drug manufacturer under close supervision by the FDA, sources said.
If the “conditional” approval designation is established, it would mean that such drugs could be obtained just like any other prescription drug. It would also mean that they would be covered by medical insurance, because they would no longer be considered experimental.
“The idea is not to compromise the notion of consumer protection but use flexible mechanisms to meet standards that are tailored to the particular circumstances,” said Dr. Lawrence C. Horowitz, a member of a federal advisory committee that is studying the mission of the FDA.
The committee’s subcommittee on human drugs and biologics, which Horowitz heads, recently recommended that the FDA develop such a conditional approval designation that would be linked to a program of post-market surveillance.
“With the explosion of science and the changing nature of the world that FDA regulates, it’s important to have a variety of tools available,” Horowitz said. “Now, in 1991, not all drugs are alike. Therefore, the appropriate way of regulating them is not necessarily the same. Consumer protection can be maintained in a variety of ways.”
If such a conditional approval policy is adopted, the first drug most likely to benefit is the experimental antiviral AIDS drug DDI, or dideoxynosine, which is already available under special expanded access programs but which has not been approved for marketing. Thus far, the only antiviral AIDS drug in the marketplace is AZT.
Sources said Kessler is “intrigued” by the proposal and that there is considerable support within the federal research community. But others in the AIDS community have expressed some reservations about the idea, saying they fear that a prolonged public debate over the concept could slow down the regular approval process of drugs already in the pipeline, particularly of DDI.
“There are going to be some concerns about whether this approach is going too far in the direction of deregulation,” said Jeff Levi, an AIDS policy consultant. “I think the AIDS drugs we are talking about can be approved under existing authority without relaxing the rules.”
Among the issues the panel will have to address is what research criteria to apply to a drug in determining whether it should be conditionally approved. Currently, drugs in such diseases as cancer and AIDS are often measured by so-called “end points,” such as survival time.
But, under possible revisions of the rules, another standard could be so-called “surrogate markers,” early indications of the drug’s effect. In AIDS, for example, a surrogate marker could be the maintenance of or an increase in the number of T4 cells, the immune system cells that are the primary target of the human immunodeficiency virus. In cancer, it could mean the shrinkage of a tumor, rather than prolonged survival or the tumor’s disappearance.
Presumably, the use of surrogate markers could be a standard used to judge a drug for approval under this category, with flexibility later on to determine if the drug has indeed provided positive clinical benefits.
